Trial Outcomes & Findings for Staccato Loxapine in Migraine (in Clinic) (NCT NCT00489476)

Last Updated: 2017-07-06

Results Overview

The primary efficacy endpoint was Pain-relief response as defined by the International Headache Society (Pain-IHS) as a pain severity of NONE or MILD. Intent to treat (ITT) with last observation carried forward (LOCF)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

168 participants

Primary outcome timeframe

Baseline and 2 h post-dose

Results posted on

2017-07-06

Participant Flow

Participant milestones

Participant milestones
Measure	Staccato Placebo Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine 5 mg ADASUVE, single dose Staccato Loxapine
Overall Study STARTED	39	43	43	43
Overall Study COMPLETED	39	42	43	43
Overall Study NOT COMPLETED	0	1	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Staccato Placebo Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine 5 mg ADASUVE, single dose Staccato Loxapine
Overall Study Lost to Follow-up	0	1	0	0

Baseline Characteristics

Staccato Loxapine in Migraine (in Clinic)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Staccato Placebo n=39 Participants Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine n=43 Participants 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine n=43 Participants 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine n=43 Participants 5 mg ADASUVE, single dose Staccato Loxapine	Total n=168 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Age, Categorical Between 18 and 65 years	39 Participants n=5 Participants	43 Participants n=7 Participants	43 Participants n=5 Participants	43 Participants n=4 Participants	168 Participants n=21 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants
Age, Continuous	42.2 years STANDARD_DEVIATION 10.94 • n=5 Participants	41.7 years STANDARD_DEVIATION 13.09 • n=7 Participants	38.5 years STANDARD_DEVIATION 11.89 • n=5 Participants	42.3 years STANDARD_DEVIATION 12.68 • n=4 Participants	41.1 years STANDARD_DEVIATION 2.20 • n=21 Participants
Sex: Female, Male Female	29 Participants n=5 Participants	38 Participants n=7 Participants	32 Participants n=5 Participants	35 Participants n=4 Participants	134 Participants n=21 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	5 Participants n=7 Participants	11 Participants n=5 Participants	8 Participants n=4 Participants	34 Participants n=21 Participants
Region of Enrollment United States	39 participants n=5 Participants	43 participants n=7 Participants	43 participants n=5 Participants	43 participants n=4 Participants	168 participants n=21 Participants

PRIMARY outcome

Timeframe: Baseline and 2 h post-dose

Population: ITT Population with LOCF

Outcome measures

Outcome measures
Measure	Staccato Placebo n=39 Participants Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine n=43 Participants 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine n=43 Participants 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine n=43 Participants 5 mg ADASUVE, single dose Staccato Loxapine
Pain-relief Response (Pain Severity of NONE or MILD) at 2 Hours	20 participants	29 participants	34 participants	33 participants

SECONDARY outcome

Timeframe: Baseline and 2 h post-dose

Population: ITT with LOCF Population

Pain-free (Pain-IHS) at the 2 hour time point

Outcome measures

Outcome measures
Measure	Staccato Placebo n=39 Participants Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine n=43 Participants 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine n=43 Participants 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine n=43 Participants 5 mg ADASUVE, single dose Staccato Loxapine
Pain-free at 2 Hours	3 Participants	12 Participants	13 Participants	9 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline through 24 h post-dose

Population: ITT with LOCF Population

The percentages of patients with sustained freedom from pain (pain-free at 2 hours after dosing with no rescue medication and no recurrence of headache from 2 to 24 hours)

Outcome measures

Outcome measures
Measure	Staccato Placebo n=39 Participants Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine n=43 Participants 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine n=43 Participants 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine n=43 Participants 5 mg ADASUVE, single dose Staccato Loxapine
Responders, Sustained Freedom From Pain	3 Participants	9 Participants	11 Participants	7 Participants

Adverse Events

Staccato Placebo

Serious events: 0 serious events

Other events: 14 other events

Deaths: 0 deaths

1.25 mg Staccato Loxapine

Serious events: 0 serious events

Other events: 18 other events

Deaths: 0 deaths

2.5 mg Staccato Loxapine

Serious events: 0 serious events

Other events: 21 other events

Deaths: 0 deaths

5 mg Staccato Loxapine

Serious events: 0 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Staccato Placebo n=39 participants at risk Staccato Placebo, 0 mg Staccato Placebo: Placebo aerosol inhalation (0mg)	1.25 mg Staccato Loxapine n=43 participants at risk 1.25 mg ADASUVE, single dose Staccato Loxapine	2.5 mg Staccato Loxapine n=43 participants at risk 2.5 mg ADASUVE, single dose Staccato Loxapine	5 mg Staccato Loxapine n=43 participants at risk 5 mg ADASUVE, single dose Staccato Loxapine
Gastrointestinal disorders Dry Mouth	5.1% 2/39 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	4.7% 2/43 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	4.7% 2/43 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Gastrointestinal disorders Dysgeusia	12.8% 5/39 • Number of events 5 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	18.6% 8/43 • Number of events 8 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	23.3% 10/43 • Number of events 10 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	37.2% 16/43 • Number of events 16 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Gastrointestinal disorders Hypoaesthesia Oral	0.00% 0/39 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	4.7% 2/43 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Gastrointestinal disorders Oral Discomfort	2.6% 1/39 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	7.0% 3/43 • Number of events 3 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
General disorders Fatigue	7.7% 3/39 • Number of events 3 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	7.0% 3/43 • Number of events 3 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	14.0% 6/43 • Number of events 6 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Nervous system disorders Disturbance in Attention	0.00% 0/39 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	4.7% 2/43 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Nervous system disorders Dizziness	5.1% 2/39 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	7.0% 3/43 • Number of events 3 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Nervous system disorders Somnolence	12.8% 5/39 • Number of events 5 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	4.7% 2/43 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	23.3% 10/43 • Number of events 10 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	23.3% 10/43 • Number of events 10 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Respiratory, thoracic and mediastinal disorders Pharyngeal Hypoaesthesia	0.00% 0/39 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	7.0% 3/43 • Number of events 3 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Respiratory, thoracic and mediastinal disorders Throat Irritation	0.00% 0/39 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	7.0% 3/43 • Number of events 3 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff
Vascular disorders Hypotension	2.6% 1/39 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	0.00% 0/43 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	2.3% 1/43 • Number of events 1 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff	4.7% 2/43 • Number of events 2 • Adverse events (AEs) were considered treatment related from the first exposure to study treatment until 30 days after the last treatment Adverse events (AEs) were assessed at 9 pre-specified time points as well as whenever spontaneously reported by the subjects or study staff

Additional Information

Executive VP, Research & Development, Regulatory & Quality

Alexza Pharmaceuticals, Inc

Phone: 650.944.7071

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60